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在一家三级垂体中心生化指标未得到控制的肢端肥大症患者的长期治疗结果

Long-term treatment outcomes of acromegaly patients presenting biochemically-uncontrolled at a tertiary pituitary center.

作者信息

Carmichael John D, Broder Michael S, Cherepanov Dasha, Chang Eunice, Mamelak Adam, Said Qayyim, Neary Maureen P, Bonert Vivien

机构信息

Pituitary Center, Cedars-Sinai Medical Center, 8700 Beverly Blvd, Los Angeles, CA, 90048, USA.

Partnership for Health Analytic Research, LLC, 280 S. Beverly Dr., Suite 404, Beverly Hills, CA, 90212, USA.

出版信息

BMC Endocr Disord. 2017 Aug 4;17(1):49. doi: 10.1186/s12902-017-0199-x.

Abstract

BACKGROUND

Acromegaly is a rare, slowly progressive disorder resulting from excessive growth hormone (GH) production by a pituitary somatotroph tumor. The objective of this study was to examine acromegaly treatment outcomes during long-term care at a specialized pituitary center in patients presenting with lack of biochemical control.

METHODS

Data came from an acromegaly registry at the Cedars-Sinai Medical Center Pituitary Center (center). Acromegaly patients included in this study were those who presented biochemically-uncontrolled for care at the center. Biochemical control status, based on serum insulin-like growth factor-1 values, was determined at presentation and at study end. Patient characteristics and acromegaly treatments were reported before and after presentation by presenting treatment status and final biochemical control status. Data on long-term follow-up were recorded from 1985 through June 2013.

RESULTS

Seventy-four patients presented uncontrolled: 40 untreated (54.1%) and 34 (45.9%) previously-treated. Mean (SD) age at diagnosis was 43.2 (14.7); 32 (43.2%) were female patients. Of 65 patients with tumor size information, 59 (90.8%) had macroadenomas. Prior treatments among the 34 previously-treated patients were pituitary surgery alone (47.1%), surgery and medication (41.2%), and medication alone (11.8%). Of the 40 patients without prior treatment, 82.5% achieved control by study end. Of the 34 with prior treatment, 50% achieved control by study end.

CONCLUSIONS

This observational study shows that treatment outcomes of biochemically-uncontrolled acromegaly patients improve with directed care, particularly for those that initially present untreated. Patients often require multiple modalities of treatment, many of which are offered with the highest quality at specialized pituitary centers. Despite specialized care, some patients were not able to achieve biochemical control with methods of treatment that were available at the time of their treatment, showing the need for additional treatment options.

摘要

背景

肢端肥大症是一种罕见的、进展缓慢的疾病,由垂体生长激素细胞肿瘤分泌过多生长激素(GH)所致。本研究的目的是在一家专业垂体中心对生化控制不佳的患者进行长期护理期间,考察肢端肥大症的治疗效果。

方法

数据来自雪松西奈医疗中心垂体中心(该中心)的肢端肥大症登记处。本研究纳入的肢端肥大症患者为那些在该中心接受生化控制不佳护理的患者。根据血清胰岛素样生长因子-1值确定生化控制状态,在就诊时和研究结束时进行测定。按就诊时的治疗状态和最终生化控制状态报告患者特征和肢端肥大症治疗情况。记录了1985年至2013年6月的长期随访数据。

结果

74例患者生化控制不佳:40例未接受过治疗(54.1%),34例(45.9%)曾接受过治疗。诊断时的平均(标准差)年龄为43.2(14.7)岁;32例(43.2%)为女性患者。在65例有肿瘤大小信息的患者中,59例(90.8%)患有大腺瘤。34例曾接受过治疗的患者之前的治疗方式为单纯垂体手术(47.1%)、手术加药物治疗(41.2%)和单纯药物治疗(11.8%)。在40例未接受过治疗的患者中,82.5%在研究结束时实现了控制。在34例曾接受过治疗的患者中,50%在研究结束时实现了控制。

结论

这项观察性研究表明,生化控制不佳的肢端肥大症患者通过针对性护理,治疗效果会得到改善,尤其是那些最初未接受治疗的患者。患者通常需要多种治疗方式,其中许多在专业垂体中心能以最高质量提供。尽管有专业护理,但一些患者在接受治疗时采用的现有治疗方法未能实现生化控制,这表明需要更多治疗选择。

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